Pharmaceutical Technology

FEBRUARY 14, 2023

It will also support smaller dose vials development, selection of strain as per the US Food and Drug Administration (FDA) recommendations, and transition to the commercial market. The company stated that the deal would maintain the access of the US public to its vaccine.

Vaccination 162

Vaccination Vaccine Protein Engineer 162

Pharmaceutical Technology

JULY 7, 2022

Genetic mutations, both germline and acquired, are behind a large proportion of the most debilitating and sometimes life-threatening human diseases. But scientists have struggled to find effective treatments for many of these diseases since the dawn of modern medicine. A new frontier in cancer research.

Gene Therapy 245

Gene Therapy Gene Medicine Genetics 245

Roots Analysis

JUNE 1, 2022

DNA Cloning Kit Providers – Current Market Landscape. The market is highly fragmented, featuring the presence of both new entrants and established players, based in different geographical regions. DNA Cloning Kits Market. DNA Cloning Reagent Providers – Current Market Landscape. DNA Cloning Reagents Market.

DNA 52

DNA Genetics Reagent Marketing 52

Pharmaceutical Technology

JANUARY 19, 2023

Nuvaxovid is a protein-based vaccine created from the genetic sequence of the initial SARS-CoV-2 virus strain. Novavax president and CEO Stanley Erck said: “We are pleased to collaborate with SK bioscience to offer our protein-based vaccine, Nuvaxovid, for use as a booster in adults regardless of previous vaccine history.

Vaccination 147

Vaccination Vaccine Immune Response Protein 147

Pharmaceutical Technology

SEPTEMBER 22, 2022

AVR-RD-04 works by genetically modify a patient's hematopoietic stem cells (HSCs) to express the gene that encodes the cystinosin protein. Cystinosis patients have a high deficiency of this protein. A rare and fatal ailment, cystinosis is characterised by cystine buildup in cellular organelles called lysosomes.

Gene Therapy 147

Gene Therapy Gene Protein Clinical Trials 147

pharmaphorum

SEPTEMBER 14, 2020

Merck & Co has signed a multi-billion cancer drug development deal, which will see it investing $1 billion in partner Seattle Genetics. US-based Merck, known as MSD outside North America, will globally develop and market Seattle’s investigational antibody-drug conjugate (ladiratuzumab vedotin). billion equity investment in 5.0

Genetics 52

Genetics Development Drugs Protein 52

Pharmaceutical Technology

JUNE 13, 2023

Ziftomenib targets the menin-KMT2a protein-protein interaction that may occur in patients with KMT2ar or NPM1m genetic alterations. The aberrant protein complex is essential for regulating leukaemia-promoting gene expression. Thus, targeting menin may be an effective strategy for patients with high unmet needs.

Gene Expression 130

Gene Expression Protein Sales Genetics 130

Outsourcing Pharma

JUNE 7, 2023

Duchenne Muscular Dystrophy (DMD) is a genetic disorder that primarily affects males, caused by a mutation in the gene that encodes for a protein called dystrophin. This protein is crucial for the normal functioning of muscles, and its absence leads to progressive muscle weakness and wasting.

Protein 82

Protein Genetics Gene Research 82

pharmaphorum

APRIL 23, 2021

billion in milestone payments in the deal, which will focus on using small-molecule compounds to “fine tune” the effects of genetic medicines in the body. The approach relies on the use of drug responsive domains (DRDs) on the gene-editing medicine, which can be used to either increase or decrease protein expression.

Gene Editing 52

Gene Editing Genetics Drugs Gene 52

Pharmaceutical Technology

AUGUST 17, 2022

Merck (MSD outside North America) has entered a partnership agreement with Orna Therapeutics for discovering, developing and marketing various programmes based on next-generation RNA technology. These programmes will include therapies and vaccines in infectious disease and oncology areas.

RNA 147

RNA Vaccination Vaccine In-Vivo 147

pharmaphorum

NOVEMBER 7, 2022

This month, we highlight new appointments from Lumeris, Lineage Cell Therapeutics, 1910 Genetics, and ADC Therapeutics. . As we continue to enter new markets, grow within our existing markets and reach a broader population, we continue to invest in our operations, talent and technology capabilities to support that growth.”

Life Science 105

Life Science Genetics Business Development Marketing 105

Pharmaceutical Technology

MAY 25, 2023

DMD is a rare genetic disorder caused by changes to the dystrophin protein, leading to progressive muscular degeneration. The FDA has told Sarepta that in this additional timeframe, it will complete final label negotiations and work out post-marketing commitments.

Gene Therapy 130

Gene Therapy Gene Clinical Trials Clinical Trials 130

pharmaphorum

OCTOBER 2, 2020

The rolling review, which is designed to speed up the assessment of a promising vaccine or drug by the EMA’s CHMP scientific committee, will continue until there is enough evidence available to support a marketing authorisation. But this does not mean that the CHMP is ready to make a conclusion on the safety and efficacy of the vaccine.

Regulation 105

Regulation Vaccination Vaccine Protein 105

Pharmaceutical Technology

MARCH 1, 2023

“By combining Tevard’s ability to restore the production of critical proteins with Vertex’s clinical, regulatory, and manufacturing expertise, we hope to make an important difference for patients and their families.” The company is pioneering tRNA-based therapeutics for modulating mRNA function and curing several genetic diseases.

Development 130

Development Gene Therapy Genetics Genetic Disease 130

pharmaphorum

FEBRUARY 16, 2021

Inflammasomes are a group of intracellular proteins associated with the inflammatory response, and prior research has linked the NLRP3 inflammasome to the aggregation of amyloid beta and tau protein – which together form the characteristic plaques and tangles in the brains of AD patients.

Research 119

Research Drugs Protein Development 119

XTalks

FEBRUARY 19, 2025

It occurs when a genetic error causes the overproduction of a protein called CSF1, which attracts inflammatory cells to the joint, often causing pain, swelling and reduced mobility. In 2024, Deciphera was acquired by ONO Pharmaceutical for $2.4 TGCT is a rare, non-cancerous tumor that develops inside or near joints.

FDA Approval 59

FDA Approval Drugs Trials Clinical Trials 59

Roots Analysis

JANUARY 19, 2024

Introduction In a strategic move to accelerate the development and launch of genetic medicines, BridgeBio Pharma has successfully secured up to USD 1.25 ATTR is an inherited disease causing abnormal transthyretin protein and the accumulation of amyloid protein in various tissues, including the heart.

Genetics 40

Genetics Medicine Development Protein 40

XTalks

MARCH 7, 2024

Industry professionals will explore the market, innovative formulations and nutritional solutions tailored to empower women toward optimal health and well-being. The test accurately identified a range of genetic abnormalities, including trisomies and microdeletions.

Genetics 115

Genetics Hormones Genome Genomics 115

Pharmaceutical Technology

MAY 3, 2023

The US Food and Drug Administration (FDA) has granted Tenaya Therapeutics’ gene therapy TN-201 a fast track designation for the treatment of myosin binding protein C3 (MYBPC3)-associated hypertrophic cardiomyopathy (HCM). Tenaya’s treatment delivers a functional MYPC3 gene, restoring myosin-binding protein levels to normal.

Gene Therapy 242

Gene Therapy Gene Protein Trials 242

XTalks

JULY 22, 2021

Impossible Foods announced that they would enter the vegan chicken market later this year by introducing their own plant-based Impossible Chicken Nuggets. This already crowded market will now include more competition with the entry of Impossible Foods. In 2021, the global poultry market is predicted to grow at a CAGR of 3.8

Marketing 98

Marketing Production Genetics Branding 98

Delveinsight

JULY 23, 2020

The current Familial chylomicronemia syndrome market is mainly dominated by traditional TG-lowering agents, and as per DelveInsight analysis, the market worth was estimated to be USD 1.0 Familial Chylomicronemia Syndrome Market. Similar is the case with Japan FCS market. million in 2017. 2017, Stroes et al.

Marketing 61

Marketing Gene Therapy Drugs Genome 61

Pharmaceutical Technology

JANUARY 22, 2023

Elsewhere, Albireo Pharma is aiming to file for supplemental NDA to the FDA for its drug Bylvay (odevixibat), which could be the second treatment for Alagille syndrome, a rare genetic disorder caused by a JAG1 gene mutation. The condition is characterised by blockage of the flow of bile from the liver, and pruritus or itching.

Drugs 317

Drugs Gene Hormones Clinical Trials 317

STAT News

NOVEMBER 23, 2022

The one-time treatment, called Hemgenix, was developed by the Dutch biotech company UniQure and will be marketed by CSL Behring, an Australian pharmaceutical company. Hemgenix will cost $3.5 million, making it the most expensive drug approved to date. Continue to STAT+ to read the full story…

Gene Therapy 97

Gene Therapy FDA Approval Gene Genetics 97

Roots Analysis

MARCH 16, 2023

It is important in the process of protein synthesis because mRNA is responsible for transferring genetic information from DNA to ribosomes, which then decodes the genetic information into a protein. What are the key trends that are shaping up the mRNA therapeutics and vaccines market?

Vaccination 52

Vaccination Vaccine DNA Protein 52

Delveinsight

JANUARY 18, 2021

Angelman syndrome (AS) is a complex genetic rare disorder that affects the nervous system. The present Angelman syndrome treatment market revolves around the management of symptoms rather than curing the condition. . However, the journey to secure the FDA nod in the Angelman syndrome market landscape does not seem to be all flowery.

Marketing 52

Marketing Gene Drugs Protein 52

XTalks

JANUARY 3, 2024

In individuals affected by ATTR, which includes both hereditary and wild-type (non-hereditary) variants, the TTR protein forms fibrils that accumulate in various tissues. These tissues comprise peripheral nerves, the heart, gastrointestinal system, eyes, kidneys, central nervous system, thyroid and bone marrow.

FDA Approval 105

FDA Approval Protein RNA Clinical Trials 105

pharmaphorum

DECEMBER 1, 2020

The filing for a conditional marketing authorisation completed the rolling submission process that began on October 6. They work by using a short length of genetic code that makes the body produce the “Spike” protein seen on the surface of the SARS-CoV-2 coronavirus.

Vaccination 88

Vaccination Vaccine RNA Clinical Trials 88

Delveinsight

SEPTEMBER 3, 2021

Further, there exist no curative therapies in the Charcot-Marie-Tooth disease market. The present CMT disease therapeutics market is purely based on supportive and symptomatic management that includes occupational therapies, physiotherapy, ankle-foot orthoses (AFO), and shoe inserts. The condition has no cure at hand.

Pharma Companies 52

Pharma Companies Marketing Clinical Trials Clinical Trials 52

XTalks

APRIL 3, 2024

The RBCs in circulation are marked by proteins in the complement system for removal by the spleen and liver. While Soliris had its market launch 17 years ago with $3.2 In 2023, its second year on the market, the drug earned $91 million in sales. AstraZeneca is also venturing into cell therapy and genetic medicine.

FDA Approval 102

FDA Approval Sales Trials Development 102

pharmaphorum

OCTOBER 4, 2022

AavantiBio, a precision genetic medicine company that was bankrolled by Solid’s rival Sarepta back in 2020, focuses on neuromuscular and cardiac rare diseases. SGT-003 utilises a novel muscle-tropic AAV capsid (AAV-SLB101) for delivery of Solid’s proprietary and differentiated neuronal nitric oxide synthase (nNOS) microdystrophin protein.

Gene Therapy 115

Gene Therapy Gene Life Science Genetics 115

pharmaphorum

MARCH 17, 2021

million shares at $20-$22 each would give the company a market cap of around $1.8 However the shares are already trading significantly higher than this, touching nearly $37 at one point in pre-market trading following favourable early stage clinical trial data announced earlier this week.

Clinical Trials 72

Clinical Trials Clinical Trials Trials Marketing 72

pharmaphorum

AUGUST 23, 2021

DNA vaccines work by delivering a genetically-engineered plasmid containing the DNA sequence encoding the desired antigen – in this case the SARS-CoV-2 spike protein – which is then taken up by cells.

DNA 98

DNA Vaccination Vaccine Genetic Engineering 98

pharmaphorum

FEBRUARY 5, 2021

J&J’s vaccine works by using an adenovirus as vector to deliver the genetic code of the Spike protein found on the coronavirus to the body. This causes the body to make the Spike protein, then produce antibodies against it that confer protection if virus infection occurs after the vaccination.

Vaccination 84

Vaccination Vaccine Protein Medicine 84

pharmaphorum

AUGUST 1, 2022

The US biotech – which already has three antisense drugs for DMD on the market – had said earlier it was planning to delay the application into 2023, so the new announcement marks an acceleration of its plans.

Gene Therapy 105

Gene Therapy Gene Genetics Trials 105

pharmaphorum

OCTOBER 3, 2022

DMD is a severe, progressive muscle-wasting genetic condition caused by the lack of a protein called dystrophin. The re-evaluation followed a six-year period during which the DMD drug had been available under a managed access agreement (MAA), which ends in January next year, and under which 60 children have had access to Translarna.

Gene Therapy 98

Gene Therapy Gene FDA Approval DNA 98

pharmaphorum

SEPTEMBER 30, 2022

Roche-partnered SRP-9001 (delandistrogene moxeparvovec) has been submitted for approval to treat ambulatory (walking) patients with DMD, a genetic disorder characterised by progressive muscle degeneration due to alterations in a protein called dystrophin that helps keep muscle cells intact.

Gene Therapy 105

Gene Therapy Gene Genetics Protein 105

pharmaphorum

AUGUST 7, 2020

Biogen has bought a licence to co-develop and co-market potential Parkinson’s disease drugs with US biotech Denali, in a deal worth more than $2.1 Biogen and Denali will co-market the lead LRRK2 product in the US and China, and Biogen will commercialise in all other markets.

Bacteria 69

Bacteria Marketing Licensing Licensing 69